CA3069150A1 - Methods and compositions for treating a bleeding event in a subject having hemophilia - Google Patents

Methods and compositions for treating a bleeding event in a subject having hemophilia Download PDF

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Publication number
CA3069150A1
CA3069150A1 CA3069150A CA3069150A CA3069150A1 CA 3069150 A1 CA3069150 A1 CA 3069150A1 CA 3069150 A CA3069150 A CA 3069150A CA 3069150 A CA3069150 A CA 3069150A CA 3069150 A1 CA3069150 A1 CA 3069150A1
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CA
Canada
Prior art keywords
subject
less
nucleotides
hemophilia
effective amount
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3069150A
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English (en)
French (fr)
Inventor
Akin Akinc
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
Original Assignee
Genzyme Corp
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Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CA3069150A1 publication Critical patent/CA3069150A1/en
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CA3069150A 2017-07-10 2018-07-10 Methods and compositions for treating a bleeding event in a subject having hemophilia Pending CA3069150A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201762530518P 2017-07-10 2017-07-10
US62/530,518 2017-07-10
US201762599223P 2017-12-15 2017-12-15
US62/599,223 2017-12-15
US201862614111P 2018-01-05 2018-01-05
US62/614,111 2018-01-05
US201862673424P 2018-05-18 2018-05-18
US62/673,424 2018-05-18
PCT/US2018/041400 WO2019014187A1 (en) 2017-07-10 2018-07-10 METHODS AND COMPOSITIONS FOR TREATING A BLEEDING EVENT IN A SUBJECT WITH HEMOPHILIA

Publications (1)

Publication Number Publication Date
CA3069150A1 true CA3069150A1 (en) 2019-01-17

Family

ID=63036452

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3069150A Pending CA3069150A1 (en) 2017-07-10 2018-07-10 Methods and compositions for treating a bleeding event in a subject having hemophilia

Country Status (17)

Country Link
US (2) US20200163987A1 (https=)
EP (3) EP4588512A3 (https=)
JP (3) JP2020530442A (https=)
KR (2) KR20250159269A (https=)
CN (1) CN111093771A (https=)
AU (2) AU2018301801A1 (https=)
BR (1) BR112020000400A2 (https=)
CA (1) CA3069150A1 (https=)
CO (1) CO2020001354A2 (https=)
IL (1) IL271646A (https=)
MX (1) MX2025007819A (https=)
NZ (1) NZ761103A (https=)
PY (1) PY1855437A (https=)
SG (1) SG11201912243PA (https=)
TW (2) TW202434265A (https=)
UY (1) UY37803A (https=)
WO (1) WO2019014187A1 (https=)

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WO2022076835A1 (en) * 2020-10-09 2022-04-14 Dyax Corp. Methods and apparatuses for modeling adamts13 and von willebrand factor interactions
IL303452A (en) * 2020-12-06 2023-08-01 Genzyme Corp Treatment of hemophilia with fitusiran
WO2022120291A1 (en) * 2020-12-06 2022-06-09 Genzyme Corporation Treatment of hemophilia with fitusiran
KR102755621B1 (ko) 2021-08-19 2025-01-21 (주) 바텍에스이씨 폭발 위험도 산출 장치 및 방법
KR102826316B1 (ko) 2021-08-19 2025-06-27 (주) 바텍에스이씨 액체 누설에 의한 폭발 위험도 산출 장치 및 방법
TW202342065A (zh) 2021-12-22 2023-11-01 美商健臻公司 治療血友病的方法和組成物
IL317440A (en) 2022-06-08 2025-02-01 Genzyme Corp FITUSIRAN for the treatment of hemophilia A and B
WO2023240193A2 (en) 2022-06-08 2023-12-14 Genzyme Corporation Treatment of hemophilia with fitusiran in pediatric patients
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WO2024094705A1 (en) 2022-10-31 2024-05-10 Sanofi Drug delivery device having a two-part user indicator
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EP4611851A1 (en) 2022-10-31 2025-09-10 Sanofi Drug delivery device with feedback element and method for providing a user of a drug delivery device feedback regarding a dose dispensing process
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CN120129547A (zh) 2022-10-31 2025-06-10 赛诺菲 用于药物递送装置的装置本体、用于药物递送装置的组件以及药物递送装置
EP4611846A1 (en) 2022-10-31 2025-09-10 Sanofi Plunger for expelling a drug, drug delivery device, rear sub-assembly and corresponding methods
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EP4612687A1 (en) 2022-11-02 2025-09-10 Genzyme Corporation Systems and methods for modeling thrombin-antithrombin
CN121285406A (zh) 2023-06-05 2026-01-06 赛诺菲 药物递送装置
EP4719537A1 (en) 2023-06-05 2026-04-08 Sanofi Drug delivery device
EP4719527A1 (en) 2023-06-05 2026-04-08 Sanofi Drug delivery device
WO2024251797A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device
CN121240902A (zh) 2023-06-05 2025-12-30 赛诺菲 自驱动药剂容器和药剂递送装置
CN121240901A (zh) 2023-06-05 2025-12-30 赛诺菲 具有侧向柔性针头套筒的药物递送装置
WO2024251796A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device having two drug delivery arrangements
EP4719538A1 (en) 2023-06-05 2026-04-08 Sanofi Drug delivery device
EP4719531A1 (en) 2023-06-05 2026-04-08 Sanofi Drug delivery device and method for decoupling a drug administration element and at least one part of a drug delivery device
WO2024251792A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device
CN121263160A (zh) 2023-06-05 2026-01-02 赛诺菲 刺孔封闭辅助构件、药物递送装置和方法
CN121816187A (zh) 2023-09-15 2026-04-07 建新公司 用非妥西兰治疗血友病a
WO2025162927A1 (en) 2024-01-29 2025-08-07 Sanofi Purifying device, system and method for purifying a needle for delivering a medicament
WO2025162926A1 (en) 2024-01-29 2025-08-07 Sanofi Needle arrangement, drug delivery device comprising the needle arrangement and method for operating the drug delivery device
WO2025162928A1 (en) 2024-01-29 2025-08-07 Sanofi Needle shroud unlock device for a medicament delivery device
WO2025171279A1 (en) 2024-02-09 2025-08-14 Genzyme Corporation Treatment of hemophilia with fitusiran
WO2025212516A1 (en) 2024-04-01 2025-10-09 Genzyme Corporation Treatment of factor x deficiency with fitusiran
CN118557756B (zh) * 2024-05-24 2025-04-29 华中科技大学同济医学院附属协和医院 一种基于aav的遗传性抗凝血酶缺乏的基因疗法
WO2026012399A1 (zh) * 2024-07-10 2026-01-15 正大天晴药业集团股份有限公司 靶向serpinc1的双链核糖核酸

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